Apertura Gene Therapy
Apertura Gene Therapy is a biotechnology company that treats debilitating diseases with limited options.
- Company typePrivate
- Founded2021
- HeadquartersNewyork, United Kingdom
- Headcount11–50
- GTM typeB2B
- OfferingServices
What Apertura Gene Therapy does
Apertura Gene Therapy offers treatment options for debilitating diseases with few options. It is in a unique position to develop genetic medicines by simultaneously engineering AAV capsids, regulatory elements, and payloads to overcome current gene therapy limitations. They are dedicated to the advancement of gene therapy and believe that by collaborating with corporate and academic partners, patients, and disease.
Apertura Gene Therapy firmographics
Firmographics- Name
- Apertura Gene Therapy
- Legal name
- Apertura Gene Therapy
- Website
- https://aperturagtx.com/
- Company type
- Private
- Founded year
- 2021
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Apertura Gene Therapy is a biotechnology company that treats debilitating diseases with limited options.
- Ownership category
- akta.pro rank
Apertura Gene Therapy industry classification
Industry- Product category
- Gene Therapy Technology / AAV Capsid Platform
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)
- SIC
- Services-Commercial Physical & Biological Research (8731), Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)
- akta.pro secondary industries
- Neurology/CNS Gene Therapies (HLAAACAH), Gene Therapy (Viral Vector/Plasmid) CDMO (HLAGABAE)
Keywords
Where Apertura Gene Therapy is headquartered
LocationHeadquarters
- HQ city
- Newyork
- HQ country
- United Kingdom
- HQ region
- Europe
Offices1 record
Markets served
Apertura Gene Therapy business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure
Revenue model
- Manufacturing Services: Through partnership with Viralgen CDMO, supporting clinical manufacturing of gene therapies using TfR1 CapX for Apertura's licensees.
- Academic Access Program: Open Aperture MTA program provides no-cost access to academic researchers for non-commercial research, potentially leading to future licensing opportunities.
- Technology Licensing: Licensing of TfR1 CapX and other capsid technologies to biopharma companies and patient advocacy organizations for development of CNS gene therapies. Partners include Galibra Neuroscience, Emugen Therapeutics, AviadoBio, and others.
- Collaboration and Development Agreements: Strategic collaborations with patient advocacy organizations, academic institutions, and government agencies (NIH CRADA, ARPA-H consortium) that may include milestone payments, research funding, and development support.
Go-to-market motion1 record
Distribution channels3 records
Marketing channels5 records
Apertura Gene Therapy product offering
Product offeringCore offering
Apertura Gene Therapy develops and licenses proprietary AAV capsid technology, principally TfR1 CapX, that crosses the blood-brain barrier to enable intravenous delivery of genetic medicines to the central nervous system. The company operates a platform that combines machine-learning-guided capsid engineering, genetic regulatory element design, and payload optimization, and monetizes this platform through licensing deals, strategic collaborations, and internal gene therapy programs targeting rare CNS disorders.
Product overview
Apertura Gene Therapy is a biotechnology company developing genetic medicines through a platform approach that simultaneously engineers AAV capsids, genetic regulatory elements, and payloads. The core product is TfR1 CapX, a next-generation AAV capsid that crosses the blood-brain barrier via human transferrin receptor 1 targeting. Supporting platforms include the Fit4Function capsid engineering platform (using machine learning for multi-trait optimization) and the GRE platform for cell type-specific expression. The Open Aperture program provides academic researchers no-cost access to TfR1 CapX for non-commercial research. The company operates both internal pipeline programs (including TSC1/TSC2 programs) and partners with external organizations for various CNS disease programs.
Differentiator
Problem solved
Functional benefit
Products and services
- TfR1 CapX
- Open Aperture Program
- TSC1/TSC2 Gene Therapy Programs
Quantifiable outcome
- Over 50% neuronal transduction and 90% astrocyte transduction across multiple brain regions in preclinical studies
- +3 more outcomes
Companies that use Apertura Gene Therapy
Customer profileNamed customers8 records
Segments4 records
Ideal customer profiles4 records
Apertura Gene Therapy technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
AI capability1 record
Feature7 records
Apertura Gene Therapy partnerships and signals
Strategic signalPartnerships
14 partnerships are on record, tiered major, core and minor.
- Foundation for Angelman Syndrome Therapeutics (FAST)majorAgreement for access to TfR1 CapX for Angelman syndrome gene therapy research and development programs. FAST is the leading patient advocacy organization for Angelman syndrome, affecting approximately 1 in 15,000 people.
- ARPA-H THRIVE Program ConsortiummajorPart of consortium led by Broad Institute's Center for Therapeutic Genetics selected for ARPA-H THRIVE funding. Developing gene therapies for rare childhood diseases affecting 3M+ children. Uses TfR1 CapX with base and prime gene editing technologies. Manufacturing support from Viralgen.
- NIH (NICHD and NHGRI)majorCRADA collaboration to test gene therapy for Niemann-Pick Disease Type C1 using TfR1 CapX. Multi-year research with funding support from Ara Parseghian Medical Research Fund. Goal to advance through regulatory review and manufacturing.
- TSC AlliancemajorCollaboration to advance TSC1 and TSC2 gene therapy programs using TfR1 CapX for tuberous sclerosis complex. Leverages TSC Alliance Preclinical Consortium infrastructure. Committed to raise $1.76M over 18 months for preclinical evaluation.
- AviadoBiomajorLicensed TfR1 CapX for use with vMiX RNAi platform. Developing AVB-406 for Alzheimer's disease and other tauopathies. Headquartered in London with US operations.
- Broad Institute ScientistscoreOngoing collaboration with scientists including Ben Deverman and Sonia Vallabh for presentations at ASGCT and joint development of gene therapy approaches (prion disease, base editing strategies).
- ViralgencoreStrategic manufacturing collaboration. Viralgen provides GMP manufacturing services including plasmid production, fill-finish, and quality control testing for TfR1 CapX. Leverages its integrated AAV manufacturing platform with established production framework deployed across 1,500+ batches. Supports clinical-scale production for Apertura's licensees.
- Finding Hope for Frizzle (FRRS1L)majorLicense agreement for gene therapy development targeting FRRS1L disease (early infantile epileptic encephalopathy type 37). Parent-led nonprofit planning clinical trial in 2H 2026, pending regulatory approval.
- Galibra NeurosciencemajorLicensed TfR1 CapX for GABA-related disorders including succinic semialdehyde dehydrogenase deficiency (SSADHD). Co-founded by researchers at Boston Children's Hospital and Harvard Medical School.
- Emugen TherapeuticsmajorLicensed TfR1 CapX for neurodevelopmental and neurodegenerative disease programs. Founded by academic founders from Broad Institute, Harvard and MIT.
- Rett Syndrome Research Trust (RSRT)majorCollaboration to license TfR1 CapX for genetic medicines targeting Rett syndrome. RSRT's MECP2 Editing Consortium (six labs) will utilize the capsid under $40M Roadmap to Cures initiative targeting three genetic medicines in clinical trials by 2028.
- Whitehead InstituteminorCollaboration on prion disease gene therapy combining Apertura's TfR1 capsid with CHARM epigenetic editing platform from Jonathan Weissman's lab. Vallabh-Minikel lab at Broad leads the project.
- Broad Institute of MIT and HarvardcoreCore technology source. Company founded on platform technologies developed by Ben Deverman, Ph.D. at the Broad Institute's Vector Engineering Lab. Apertura holds exclusive rights to AAV capsids developed in the Deverman Lab. Collaborates on ongoing research and presentations at scientific conferences.
- Harvard Medical SchoolcoreTechnology source for genetic regulatory elements. Sponsored research and licensing agreement with Harvard University (through Office of Technology Development) grants Apertura exclusive access to methods of identifying cell type-specific GREs, including the PESCA platform developed by Michael Greenberg, Ph.D.
Scale indicators4 records
Recent moves6 records
Expansion highlights6 records
Apertura Gene Therapy competitors and assessment
Company assessmentDirect peers
- 4D Molecular Therapeutics (4DMT): Clinical-stage gene therapy company developing targeted AAV vectors using its Therapeutic Vector Evolution platform for tissue-specific delivery, with multiple CNS and other tissue programs in the clinic. Direct competitor in receptor-targeted AAV capsid engineering for CNS and beyond.
- Voyager Therapeutics: Clinical-stage gene therapy company developing AAV capsid engineering (TRACER platform) for CNS diseases including Parkinson's, Huntington's, and Alzheimer's. Closest direct competitor to Apertura in receptor-targeted CNS AAV capsid development.
- Dyno Therapeutics: AI-driven AAV capsid engineering company partnering with Novartis and Roche for CNS, muscle, and eye-directed gene therapies. Direct competitor using machine learning for capsid design, analogous to Apertura's Fit4Function platform.
- Taysha Gene Therapies: Clinical-stage CNS gene therapy company developing AAV-based therapies for rare monogenic neurological diseases. Comparable indication focus and AAV delivery model, though with less proprietary capsid engineering.
- Lexeo Therapeutics: Clinical-stage gene therapy company focused on CNS disorders and cardiac diseases using AAV delivery, with programs in Alzheimer's, Friedreich's ataxia, and Rett syndrome. Competes for the same biopharma partners and disease indications.
Emerging players
- Latus Bio: Early-stage gene therapy company developing AAV-based treatments for CNS disorders using novel capsid engineering. Emerging competitor with partial overlap on indication focus and delivery platform approach.
- AviadoBio: Clinical-stage AAV gene therapy company developing AVB-406 for Alzheimer's and tauopathies, currently licensing Apertura's TfR1 CapX combined with its own vMiX RNAi platform. Functions as both partner and adjacent competitor in CNS AAV gene therapy.
Broad incumbents
- Denali Therapeutics: Clinical-stage neurodegenerative disease company with proprietary BBB-crossing delivery technology (OTV platform) and broad CNS pipeline. Larger incumbent operating across multiple CNS modalities with overlapping capsid/delivery science.
- BioMarin Pharmaceutical: Established commercial-stage gene therapy company with FDA-approved CNS gene therapy (Roctavian for hemophilia A; prior CNS programs). Broad incumbent in gene therapy commercialization with adjacent delivery expertise.
- uniQure: Commercial-stage gene therapy company with FDA-approved AAV therapy (Hemgenix for hemophilia B) and CNS pipeline (Huntington's, ALS). Comparable platform-based AAV development model with broader indication footprint.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks5 records
Key highlights7 records
Customer concentration
Apertura Gene Therapy social profiles
Digital presenceApertura Gene Therapy financial estimates
Financial estimateRevenue estimate
Valuation estimate
Apertura Gene Therapy leadership team
Management profileNumber of profiles
Profiles8 records
Apertura Gene Therapy funding detail
Funding detailFunding overview
Funding rounds2 records
Investors1 record
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Apertura Gene Therapy M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Apertura Gene Therapy
What does Apertura Gene Therapy do?
Apertura Gene Therapy develops and licenses proprietary AAV capsid technology, principally TfR1 CapX, that crosses the blood-brain barrier to enable intravenous delivery of genetic medicines to the central nervous system. The company operates a platform that combines machine-learning-guided capsid engineering, genetic regulatory element design, and payload optimization, and monetizes this platform through licensing deals, strategic collaborations, and internal gene therapy programs targeting rare CNS disorders.
Is Apertura Gene Therapy a public or private company?
Apertura Gene Therapy is a private company. It is classified as venture growth investor backed and is currently operating.
When was Apertura Gene Therapy founded?
Apertura Gene Therapy was founded in 2021. It employs 11 to 50 people.
Where is Apertura Gene Therapy based?
Apertura Gene Therapy is headquartered in Newyork, United Kingdom, in the Europe region.
How does Apertura Gene Therapy make money?
Four revenue lines are on record. Manufacturing Services are the primary driver. The others are academic Access Program, technology Licensing and collaboration and Development Agreements.
Who are Apertura Gene Therapy's main competitors?
Direct peers on record are 4D Molecular Therapeutics (4DMT), Voyager Therapeutics, Dyno Therapeutics, Taysha Gene Therapies and Lexeo Therapeutics. Emerging players are Latus Bio and AviadoBio. Broad incumbents are Denali Therapeutics, BioMarin Pharmaceutical and uniQure.
Does Apertura Gene Therapy have an API?
No public API is recorded for Apertura Gene Therapy.
What industry is Apertura Gene Therapy in?
Apertura Gene Therapy's product category is Gene Therapy Technology / AAV Capsid Platform. Its primary akta.pro industry code is HLAAACAN, Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream), with a secondary code of HLAAACAH, Neurology/CNS Gene Therapies. Its NAICS code is 541714 and its SIC code is 8731.